No significant safety or dose limiting toxicities reported
PK data from Cohorts 1 and 2 consistent with preclinical and Phase 0 trial results
BOSTON, Feb. 6, 2025 /PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that Cohort 3 of its Phase 1 clinical trial enrolled three patients and all have been dosed with TTX-MC138, its lead candidate. The Safety Review Committee monitoring the clinical trial unanimously approved opening of the third cohort based on its review of Cohort 1 and 2 safety and pharmacokinetic (PK) data and is monitoring Cohort 3 as preliminary data becomes available. The dose administered to patients in the third cohort is approximately double the dose administered in the second cohort.
Several patients in the first and second cohort remain on study for continued treatment, receiving additional doses of TTX-MC138 over cycles of approximately 28 days each. No significant safety or dose limiting toxicities have been reported in any of the trial's nine patients. Analyses of PK data and pharmacodynamic (PD) activity from Cohorts 1 and 2 is ongoing. To date, the analyses suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode's previous Phase 0 clinical trial. Specifically, results from Cohort 1 confirmed the Phase 0 observation that TTX-MC138 shows evidence of pharmacodynamic activity in the presence of high baseline expression of miR-10b, reaching a 66% inhibition at 24 hours after infusion, similar to that seen in the Phase 0 trial. Additionally, TTX-MC138 activity increased with the escalated dose administered in Cohort 2 and was consistent at subsequent administrations suggesting a favorable pharmacokinetic profile.
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode's 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.
About the Trial
TransCode's Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation phase.
Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).
About TransCode Therapeutics
TransCode is a clinical-stage oncology company focused on treating metastatic disease. The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class RNA therapeutic candidates designed to overcome the challenges of RNA delivery and thus unlock therapeutic access to a variety of novel genetic targets that could be relevant to treating a variety of cancers.
SOURCE TransCode Therapeutics, Inc.
